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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04955990




Registration number
NCT04955990
Ethics application status
Date submitted
29/06/2021
Date registered
9/07/2021
Date last updated
18/04/2023

Titles & IDs
Public title
A Study of Real-world Cohort of Pulmonary Arterial Hypertension (PAH) Participants
Scientific title
An International, Non-Drug Interventional, Real-world Cohort of PAH Patients Newly Initiating PAH Therapy With Guideline-directed Assessments of Disease Severity
Secondary ID [1] 0 0
67896062PAH4005
Secondary ID [2] 0 0
CR109007
Universal Trial Number (UTN)
Trial acronym
CARE PAH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Arterial Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PAH Therapies

Experimental: Participants with PAH - Participants with pulmonary arterial hypertension (PAH) who newly initiate any PAH therapy(ies) at the index date (date when a participant starts the first new PAH therapy after baseline assessments) in a routine clinical setting, either as first-line therapy, as replacement therapies, as concomitant with other PAH therapies, or have already been receiving macitentan 10 milligrams (mg) for at least 3 months prior to the index date. The primary data source for this study will be the medical records of each participant.


Treatment: Drugs: PAH Therapies
PAH therapies will be administered in a routine clinical setting and data will be collected from participants who newly initiate any PAH therapy(ies), or have already been receiving macitentan 10 mg for at least 3 months prior to the index date. Participants will receive PAH therapies such as Macitentan, Ambrisentan, Bosentan, Tadalafil, Sildenafil, Riociguat, Selexipag, Epoprostenol, Iloprost, Treprostinil, and Beraprost.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to all Cause Death
Timepoint [1] 0 0
Up to 6 years
Primary outcome [2] 0 0
Time to Death due to Pulmonary Arterial Hypertension (PAH) or First Hospitalization due to PAH
Timepoint [2] 0 0
Up to 6 years
Secondary outcome [1] 0 0
Time to Death due to PAH
Timepoint [1] 0 0
Up to 6 years
Secondary outcome [2] 0 0
Time to First all-cause Hospitalization
Timepoint [2] 0 0
Up to 6 years
Secondary outcome [3] 0 0
Time to First Morbidity/Mortality Event
Timepoint [3] 0 0
Up to 6 years
Secondary outcome [4] 0 0
Time to Clinical Worsening
Timepoint [4] 0 0
Up to 6 years
Secondary outcome [5] 0 0
Medical Resource Utilization
Timepoint [5] 0 0
Up to 6 years
Secondary outcome [6] 0 0
Change from Baseline in 6-minute Walk Distance (6MWD)
Timepoint [6] 0 0
Baseline up to 6 years
Secondary outcome [7] 0 0
Change from Baseline in World Health Organization (WHO) Functional Class (FC)
Timepoint [7] 0 0
Baseline up to 6 years
Secondary outcome [8] 0 0
Change from Baseline in N-terminal-pro-hormone Brain Natriuretic Peptide (NT-proBNP)
Timepoint [8] 0 0
Baseline up to 6 years
Secondary outcome [9] 0 0
Time to Worsening in WHO FC
Timepoint [9] 0 0
Up to 6 months
Secondary outcome [10] 0 0
Change from Baseline in the Number of low-risk Noninvasive Criteria Based on WHO FC, 6MWD, and NT-proBNP
Timepoint [10] 0 0
Baseline up to 6 years
Secondary outcome [11] 0 0
Time to all-cause Death Based on the Number of low-risk Noninvasive criteria Based on WHO FC, 6MWD, and NT-proBNP
Timepoint [11] 0 0
Up to 6 years
Secondary outcome [12] 0 0
Change from Baseline in Number of Participants Within Each Overall Risk Category (Low, Intermediate, or High) According to the Noninvasive Criteria
Timepoint [12] 0 0
Baseline up to 6 years
Secondary outcome [13] 0 0
Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to Registry to Evaluate Early and Long-term PAH Disease Management (REVEAL) Lite 1 Variables
Timepoint [13] 0 0
Baseline up to 6 years
Secondary outcome [14] 0 0
Change from Baseline in Number of Participants Within Each Overall Risk Category (low, Intermediate, or High) According to REVEAL Lite 2 Variables
Timepoint [14] 0 0
Baseline up to 6 years
Secondary outcome [15] 0 0
Time to all-cause Death Based on the Risk Category Determined by the Noninvasive Criteria
Timepoint [15] 0 0
Up to 6 years
Secondary outcome [16] 0 0
Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 1
Timepoint [16] 0 0
Up to 6 years
Secondary outcome [17] 0 0
Time to all-cause Death Based on the Risk Category Determined by the REVEAL Lite 2
Timepoint [17] 0 0
Up to 6 years
Secondary outcome [18] 0 0
Risk Assessment Strategies for Clinical Worsening or Death
Timepoint [18] 0 0
Up to 6 years
Secondary outcome [19] 0 0
Change from Baseline in Health-related Quality of Life (HRQoL) as Measured by Symptoms and Impact Questionnaire for Use in Clinical Practice (SYMPACT-CP) Questionnaire
Timepoint [19] 0 0
Baseline up to 6 years
Secondary outcome [20] 0 0
Change from Baseline in Health Related Quality of Life Assessed by European Quality of Life (EuroQoL) Group, 5-Dimension, 5-Level Questionnaire (EQ-5D-5L)
Timepoint [20] 0 0
Baseline up to 6 years
Secondary outcome [21] 0 0
Change from Baseline in Health-Related Quality of life Status as Assessed by 36-item Short-Form Health Survey (SF-36) v2 Acute Questionnaire
Timepoint [21] 0 0
Baseline up to 6 years
Secondary outcome [22] 0 0
Change from Baseline in PAH-specific Medication Adherence as Assessed by Morisky Medication Adherence Scale-8 (MMAS-8) Score
Timepoint [22] 0 0
Baseline up to 6 years
Secondary outcome [23] 0 0
Change from Baseline in Medication Adherence Questions of each Prescribed PAH Therapy Class
Timepoint [23] 0 0
Baseline up to 6 years
Secondary outcome [24] 0 0
Change from Baseline in PAH-specific Medication Adherence as Assessed by Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire
Timepoint [24] 0 0
Baseline up to 6 years
Secondary outcome [25] 0 0
Change from Baseline in Patient Global Assessment of Disease Severity (PGA-S) of PAH
Timepoint [25] 0 0
Baseline up to 6 years
Secondary outcome [26] 0 0
PAH Symptoms and Impact using SYMPACT-CP Questionnaire
Timepoint [26] 0 0
Baseline up to 6 years

Eligibility
Key inclusion criteria
- Symptomatic pulmonary arterial hypertension (PAH) in any PAH subtype

- PAH diagnosis confirmed by hemodynamic evaluation at rest at any time prior to or at
the index date fulfilling all of the criteria below: a) Mean pulmonary artery pressure
greater than (>) 20 millimeters of mercury (mm Hg), and b) Pulmonary artery wedge
pressure or left ventricular end diastolic pressure less than or equal to (<=) 15 mm
Hg, and c) Pulmonary vascular resistance greater than or equal to (>=) 3 Wood Units
(that is, >= 240 dynes seconds per centimeters penta [dyn·sec/cm^5])

- Participant satisfies either a or b: a) Newly initiating 1 or more PAH therapy(ies)
(as monotherapy or add-on therapy) at index date. These newly initiated PAH therapies
should not have been used within 3 months of the index date; b) Taking macitentan 10
milligrams (mg) therapy (as monotherapy or in combination) with no changes in PAH
therapy for within 3 months prior to the index date

- All mandated assessments must be performed and recorded at the baseline visit before
the initiation of the new PAH therapy at the index date or enrollment in the study.

- For the pulmonary arterial hypertension-symptoms and impact (PAH-SYMPACT) substudy
only: Participants initiating any endothelin receptor antagonist (ERA) or
phosphodiesterase-5 inhibitor therapies at index date or at therapy change must
provide consent to enroll in the optional PAH-SYMPACT substudy. Refusal to give
consent for the optional PAH-SYMPACT substudy will not exclude a participant from
participation in the main study
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Participants enrolled in any interventional clinical trial with an investigational
therapy in the 3-month period prior to index date

- Currently enrolled in an observational study sponsored or managed by a Janssen company

- Presence of moderate or severe obstructive lung disease (forced expiratory volume in 1
second [FEV1] / forced vital capacity [FVC] <70%; and FEV1 <60% of predicted after
bronchodilator administration) in participants with a known or suspected history of
significant lung disease, as documented by a spirometry test performed within 1 year
prior to screening

- Presence of moderate or severe restrictive lung disease (for example, total lung
capacity or FVC <60 percent [%] of normal predicted value) in participants with a
known or suspected history of significant lung disease, as documented by a spirometry
test performed within 1 year prior to screening

Study design
Purpose of the study
Other
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Kentucky
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New Mexico
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
Argentina
State/province [15] 0 0
Rosario
Country [16] 0 0
Brazil
State/province [16] 0 0
Belo Horizonte
Country [17] 0 0
Brazil
State/province [17] 0 0
Botucatu
Country [18] 0 0
Brazil
State/province [18] 0 0
Porto Alegre
Country [19] 0 0
Brazil
State/province [19] 0 0
Sao Paulo
Country [20] 0 0
Canada
State/province [20] 0 0
Alberta
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
China
State/province [22] 0 0
Beijing
Country [23] 0 0
China
State/province [23] 0 0
Chengdu
Country [24] 0 0
China
State/province [24] 0 0
Shanghai
Country [25] 0 0
Germany
State/province [25] 0 0
Bonn
Country [26] 0 0
Italy
State/province [26] 0 0
Milano
Country [27] 0 0
Italy
State/province [27] 0 0
Pavia
Country [28] 0 0
Italy
State/province [28] 0 0
Rome
Country [29] 0 0
Japan
State/province [29] 0 0
Okayama
Country [30] 0 0
Korea, Republic of
State/province [30] 0 0
Busan
Country [31] 0 0
Korea, Republic of
State/province [31] 0 0
Incheon
Country [32] 0 0
Korea, Republic of
State/province [32] 0 0
Seoul
Country [33] 0 0
Malaysia
State/province [33] 0 0
Kuala Lumpur
Country [34] 0 0
Netherlands
State/province [34] 0 0
Amsterdam
Country [35] 0 0
Netherlands
State/province [35] 0 0
Nieuwegein
Country [36] 0 0
Netherlands
State/province [36] 0 0
Rotterdam
Country [37] 0 0
Poland
State/province [37] 0 0
Kraków
Country [38] 0 0
Poland
State/province [38] 0 0
Lublin
Country [39] 0 0
Puerto Rico
State/province [39] 0 0
Guaynabo
Country [40] 0 0
Singapore
State/province [40] 0 0
Singapore
Country [41] 0 0
Spain
State/province [41] 0 0
Cardiologia
Country [42] 0 0
Spain
State/province [42] 0 0
Majadahonda
Country [43] 0 0
Spain
State/province [43] 0 0
Santander
Country [44] 0 0
Sweden
State/province [44] 0 0
Goeteborg
Country [45] 0 0
United Kingdom
State/province [45] 0 0
London
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Newcastle upon Tyne
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Papworth Everard
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Actelion
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study is designed to describe pulmonary arterial hypertension (PAH) participants in
terms of their clinical characteristics, therapies used, disease progression, and outcomes
(example, death, hospitalization, risk category for predicted mortality risk, and
patient-reported outcomes [PROs]) in real-world clinical practice. This study will collect
high-quality real-world data that may be used as a stand-alone dataset or in combination with
other studies to address relevant research questions (example, serve as an external control
dataset to another study) to support development and access to PAH therapies, as well as to
contribute to the knowledge base of PAH through publications.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04955990
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Actelion Clinical Trial
Address 0 0
Actelion
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04955990